CRISPR gene editing has been one of the important advances of the last decade, in biotechnology and increasingly in medicine. First applied to human cells in 2013, and honored with the 2020 Nobel Prize in Physiology or Medicine, its meteoric rise can make CRISPR look like the molecular equivalent of a miracle healer. But in the research and clinical trenches, CRISPR-based approaches, like any others, need to find applications where their desired effects outweigh their side effects. And finding those applications necessitates ways to identify off-target effects. Read More
Eurocine Vaccines AB has reported findings from preclinical studies evaluating its mRNA and protein technology platforms for its therapeutic herpes simplex virus type 2 (HSV-2) vaccine candidates. Initial results from the study, comparing the two technology platforms at multiple doses, demonstrate that the mRNA and the protein vaccines were well tolerated and generated T-cell and antibody responses. Read More
Inhibitors of cyclin-dependent kinase 4 (CDK4) and CDK6, such as palbociclib, have significantly improved progression-free survival of several breast cancer types, such as hormone receptor-positive, HER2-negative luminal breast cancers, with about 40% being unresponsive or refractory to therapy; the main cause of resistance is the selection of mutant clones in the target oncoprotein. Read More
Benevolentai Ltd. has submitted a clinical trial application (CTA) to the U.K.'s Medicines and Healthcare Products Regulatory Agency (MHRA) for BEN-8744, an oral, peripherally restricted, small-molecule phosphodiesterase 10 (PDE10) inhibitor in development as a first-in-class treatment for ulcerative colitis. Read More
Biocryst Pharmaceuticals Inc. has disclosed imidazole derivatives acting as activin receptor-like kinase 2 (ALK-2; ActR-IA) inhibitors reported to be useful for the treatment of diffuse intrinsic pontine glioma, spondyloarthritis and fibrodysplasia ossificans progressiva. Read More
Orgenesis Inc. and Kurve Therapeutics Inc. have announced promising preclinical study results for intranasal administration of a cell-based oncolytic virus-bearing product. Read More
Tumors have a lower interstitial pH compared to healthy tissues, with tumor acidity having emerged as a driver of tumor progression as it can lead to tumor immune evasion. Solute carrier family 4 member 4 (SLC4A4), which encodes a sodium bicarbonate cotransporter involved in pH regulation and homeostasis in normal tissue, was the focus of studies in pancreatic cancer resistance in in vivo preclinical models. Researchers tested the impact of Slc4a4 deletion in cancer cells on tumor growth, anticancer immunity and response to immunotherapy in murine models of pancreatic ductal adenocarcinoma (PDAC). Read More
Unnatural Products Inc. has synthesized cell-permeable cyclic peptides acting as E3 ubiquitin-protein ligase Mdm2 (Hdm2) and/or Mdm4 (Mdmx) inhibitors reported to be useful for the treatment of cancer. Read More
Protego Biopharma Inc. and Scripps Research Institute have identified serine/threonine kinase/endoribonuclease IRE1 (ERN1) activators reported to be useful for the treatment of diabetes, myocardial infarction, nonalcoholic fatty liver disease (NAFLD), Parkinson's disease, retinal degeneration, atherosclerosis, Gaucher disease and Alzheimer's disease, among others. Read More
Trastuzumab-based chemotherapy has demonstrated clinical benefits in the treatment of HER2+ breast cancer. There is a percentage of patients who do not respond to therapy and have a poorer prognosis than those who respond. To better understand the mechanisms behind trastuzumab resistance, researchers in China studied the role of transcription elongation factor A protein-like 9 (TCEAL9) in resistance to trastuzumab-based chemotherapy in HER+ breast cancer. Read More
Xuanzhu Pharma Co. Ltd. has described protein mono-ADP-ribosyltransferase TIPARP (PARP7; ARTD14) inhibitors reported to be useful for the treatment of cancer. Read More
Researchers from Hangzhou Healzen Therapeutics Co. Ltd. and Biopolar Hongye (Nantong) Pharmaceutical Co. Ltd. presented the discovery and preclinical characterization of a next-generation Bcl-2 inhibitor, HZ-L105, being developed for the treatment of hematological cancers. Read More
